A Study looking at the long-term use of a CFTR modulator (Ivacaftor) in children with cystic fibrosis who are less than 24 months and have a CFTR gating mutation

Details

Therapeutic category
Restore CFTR Function
Trial status
closed Participating Centres
Phase
Phase III

Full title

A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation
Trial Reference Number
105223
Trial type
Medication
Length of participation
104 weeks
Intervention
CFTR Modulator (Ivacaftor)
Last edited date
16/10/2019
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial

Who can take part?

Top inclusion criteria
  • Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
  • An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older
Top exclusion criteria
  • Subjects receiving commercially available ivacaftor treatment

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